I sighed this week when I heard, “Hey, Mom, do we have a clean bucket and some ice?”
Yes, the viral ALS ice-bucket challenge that has swept the country had reached our household as well. And though my daughter averred that she would never have heard about ALS otherwise, it pushed some of my cynic buttons. (My favorite response so far had come from an acidly hilarious Facebook friend who advised celebrities: “Just write a check to support ALS research. If you still need a gimmick and social media attention, set your hair on fire instead.”)
For me, the trouble was that I had looked into ALS research a few years ago and it had struck me then as extraordinarily frustrating. It was the field that first taught me that it’s all too common for a potential treatment to look good in initial testing and then fail to pan out when tried in a bigger clinical trial. That happened with ALS over and over again. And meanwhile, patients faced inexorable neurological degeneration and far too early deaths. (One of my most admired colleagues, Dudley Clendinen, died of ALS in 2012 after eloquently chronicling his time with it.)
But then I thought: Let’s be positive. Whatever the narcissistic elements of the ice-bucket dousings, the challenge is raising millions of dollars — more than $50 million as of Friday, from more than a million new donors, according to the ALS Association. And maybe ALS research has changed?
Indeed it has, say scientists working in the field. Not that it looks like there’s a cure around the corner, but there has been major progress of late, they say, and we can expect more to come.
“In about the last seven years, the genetics of ALS has just exploded the field, and just come up with so many new ideas for how we can tackle the disease,” said Avi Rodal, an ALS researcher at Brandeis University whose work is funded by the Blazeman Foundation for ALS.
Dr. Lucie Bruijn, chief scientist of the ALS Association that is reaping the ice-bucket windfall, also describes a field that is forging ahead in multiple directions. “The understanding of the disease, the research that has gone into it, has grown exponentially,” she says. “So we’re much closer to understanding the complexity of the disease and how to approach it in a very different way from before, when many of the trials were challenging partly because we didn’t understand the disease as closely.”
The Association, she says, is focusing on six main areas, and the ice-bucket money will likely be divided among them: “We want to invest in many areas,” she says, “to be sure not to dilute it too much but to be very strategic that we don’t put all our eggs in one basket.”
Those areas, in brief and lightly edited, as she described them:
About five to 10 percent of ALS runs through families, but 90 percent is sporadic. However, we’ve found that many of the genes we identify in familial disease are potential risk factors, and certainly also seem to be involved in the sporadic form. So there’s an underlying genetics in all ALS, but sometimes it’s more dominant than others. Continue reading